Top Tweets for #timeMatters

First off, this is an incredible day for HD patients and families. A generational, devastating, slow-bleed disease with no disease-modifying therapy now has the first ever GT in sight in the US. The rollercoaster has been real, the wait has been brutal, and today’s FDA re-alignment is long overdue. These families deserve this. I’m genuinely happy for every one of them. From a capital markets perspective, $qure is incredibly undervalued at current levels. The 3-year data that was always sufficient is now formally accepted as BLA-grade. No sham surgery — standard-of-care confirmatory control instead. RMAT added to Breakthrough and Fast Track. UK MHRA MAA filing Q3 2026, with the brand new MHRA-FDA liaison programme announced yesterday providing direct transatlantic alignment. Gulf states real. AMT-260 readout Friday now worth more from a platform validation standpoint. And Sung’s recent public comments about his patient cohort suggest the 4-year data will be compelling. Sell side will take time to update models and they need to answer “what is the US worth, what is the UK worth, what does early label access look like.” But at $81 with Prasad at CBER, no MHRA movement, and none of today’s catalysts, the market found fair value. Today’s setup is materially stronger on every dimension. The models will catch up. The compounder awakes. $qure

I truly do respect that you’re part of the rare disease community and that your family has faced something devastating. No parent should have to watch their child suffer. But I also think it’s important to recognize that rare diseases are not all ethically or clinically interchangeable. In Huntington’s disease, we are dealing with a fatal, irreversible neurodegenerative disease that progressively destroys the brain over years. There is no cure. There are no disease-modifying treatments approved to slow or stop progression. Once neurons are lost, they do not come back. Time itself becomes part of the disease burden. That changes the risk-benefit conversation. For many of us, the concern is not “science vs hope.” We want rigorous science. We want therapies that truly work. But we also recognize that requiring traditional placebo/sham-controlled pathways in every circumstance can become ethically complicated when patients are actively losing cognitive, psychiatric, and physical function in real time. Accelerated approval was created precisely because some diseases do not afford patients the luxury of waiting for perfect certainty. No one is arguing that every therapy should be approved without evidence. We are arguing that regulators should appropriately weigh: • disease severity • lack of alternatives • irreversible progression • biomarker and natural history evidence • and the reality that delay itself carries consequences For neurodegenerative diseases like HD, “wait longer for cleaner data” is not a neutral position. Patients pay for that time with pieces of their brain, independence, and future.

Some context for the UniQure $QURE post below: Earlier today, the FDA held a media call with the same "senior FDA official" who spoke to me (and other reporters) individually earlier in the week. The call was set up obstensibly so that this senior FDA official could rail against UniQure without going on the record. He did exactly this for 30 minutes, repeating the same criticisms, claiming Uniqure's Huntington’s therapy is a failure, the sham surgery would be blinded even if just small "nicks" in the skin were made in a 30-minute procedure, dismissing the idea of formally reviewing the therapy, disregarding outside scientific experts, and then accusing the media of not adequately covering what the FDA (and this official) have been doing. Reporters from Bloomberg, STAT, NY Times, WSJ, Politico and other media organizations were on the call.